Sildenafil Shows Potential for Lymphatic Malformations

Regression of congenital vascular anomalies seen in three children treated with oral sildenafil

WEDNESDAY, Jan. 25 (HealthDay News) -- Treatment with sildenafil results in regression of lymphatic malformations in children, according to three cases presented in a letter published in the Jan. 26 issue of the New England Journal of Medicine.

Glenda L. Swetman, M.D., from the Stanford University School of Medicine in California, and colleagues described three cases of regression of lymphatic malformations in children treated with oral sildenafil. Following observational results of lymphatic malformation regression in a 9-month-old girl treated with sildenafil for idiopathic pulmonary hypertension, a 12-week course of sildenafil was approved for two children with disabling lymphatic malformations.

The investigators found that in a 12-month-old boy with a lymphatic malformation involving the orbit and upper eyelid, sildenafil treatment resulted in improved eye opening, with a 25 percent increase in the ability to open the affected eye at the study end. After discontinuation of sildenafil, tissue enlargement recurred. Administration of sildenafil to a 15-month-old girl with three large lymphatic malformations, including one on her back extending over her shoulder, resulted in a 75 percent reduction in the malformations. Mild enlargement was seen four weeks following cessation of sildenafil.

"The observations described suggest that sildenafil represents an encouraging, propitious treatment for lymphatic malformations, used as monotherapy or with other treatments. A double-blind, placebo-controlled trial is under way," the authors write.

The authors disclosed involvement with a patent related to methods and compositions for treating a subject for a lymphatic malformation.